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Relapsed Acute Myeloid LeukaemiaPhase 1CARAMLNCT05942599

Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation

A Phase 1 study testing Cryopreserved BE CAR33 T Cells (BE752TBTTBCAR33PBL) in people with Relapsed Acute Myeloid Leukaemia, run by Great Ormond Street Hospital for Children NHS Foundation Trust at 1 site in United Kingdom.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial summary

In this phase 1 clinical trial, the investigators are testing an experimental medicine in children aged 6 months up to 16 years with acute myeloid leukaemia (AML), which has come back (relapsed). Read more about this study

Who is it for?
Ages 6 months to 16 years
Conditions the study is for
Relapsed Acute Myeloid Leukaemia
What is being tested?
Cryopreserved BE CAR33 T Cells (BE752TBTTBCAR33PBL)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteUnited Kingdom
Study dates
21 Jul 2023to 1 Jun 2026
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 16 years
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • Male or female patients
  • Age ranging between 6 months and <16 years
  • Medical and therapeutic criteria
  • Relapsed AML ahead of scheduled allogeneic haematopoietic stem cell transplantation (allo-SCT).
  • Morphologically confirmed with leukemic blasts in the bone marrow (>5%) or a quantifiable MRD by multiparameter flow cytometry and/or quantitative polymerase chain reaction (>10-4)
  • CD33+ leukaemia associated immunophenotype (LAIP) on >95% of blasts
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  • Eligible and fit for allogeneic hematopoietic stem cells transplantation with suitable donor available
  • Estimated life expectancy ≥ 12 weeks
  • Lansky (age < 16 years at the time of assent/consent) or performance status ≥ 70;
  • Eastern Cooperative Oncology Group ECOG performance status < 2.

Who cannot join

  • Patients/parents unwilling to undergo follow-up for 15 years
  • Foreseeable poor compliance to the study procedures
  • Evidence of disease progression after cytoreduction
  • Uncontrollable CNS leukaemia or neurological symptoms defined as CNS grade 3 (per
  • National Comprehensive Cancer Network guidelines)
  • Absence of suitable HLA matched or mismatched donor
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  • Weight < 6kgs
  • Presence of donor-specific anti-HLA antibodies directed against BE-CAR33
  • GvHD requiring systemic therapy
  • Systemic steroid therapy prednisolone >0.5mg/kg/day
  • Known hypersensitivity to test materials or related compounds
  • Active bacterial, fungal or viral infections not controlled by standard of care anti- microbial or anti-viral treatment. Uncontrolled bacteraemia/ fungaemia is defined as the ongoing detection of bacteria/fungus on blood cultures despite antibiotic or anti-fungal therapy. Uncontrolled viraemia is defined as rising viral loads on two consecutive occasions despite antiviral therapy.
  • Risk of pregnancy or non-compliance with contraception (if applicable). Girls of childbearing potential must have been tested negative in a pregnancy test within 14 days prior to inclusion.
  • Lactating female participants unwilling to stop breastfeeding
  • Prior CAR T cell therapy known to be associated with ≥Grade 3 cytokine release syndrome (CRS) or ≥Grade 3 drug-related CNS toxicity

Trial locations and contacts

1 site in 1 country.

United Kingdom1 site

  • Great Ormond Street Hospital for Children

    London, WC1N3JH

Other trials in United Kingdom

About this study

Official title: Phase 1 Study of Base Edited CAR T Cells Against AML: Deep Conditioning Ahead of Allogeneic Stem Cell Transplantation

In this phase 1 clinical trial, the investigators are testing an experimental medicine in children aged 6 months up to 16 years with acute myeloid leukaemia (AML), which has come back (relapsed). The new product is made from white blood cells (T cells) collected from a healthy donor and changed so they can kill leukaemia cells. These 'ready-made' CAR T cells have been made using a new technique called Base Editing to modify their DNA code and have been given the code name 'BE CAR-33'. This technique allows them to work after chemotherapy and also disarms them to prevent effects against normal cells. The main purpose of this study is to assess the safety of the 'BE CAR-33' therapy and to see if ready-made CAR T cells can get rid of Acute Myeloid Leukaemia ahead of a planned bone marrow transplant that will hopefully prevent the leukaemia from returning.

Read the detailed description

Who can participate? Patients aged 6 months to 16 years with relapsed acute myeloid leukaemia ahead of a planned bone marrow transplant.

What does the study involve? Patients will undergo careful screening to confirm that this treatment is adequate for them. Chemotherapy will be given prior to BE CAR-33 infusion to improve the ability of T-cells to establish and grow. Patients will then receive a single infusion of the BE CAR-33 cells. They will be closely monitored via blood and bone marrow tests for safety and to check the levels of BE CAR-33 and leukaemia cells. The investigators expect patients to be in hospital for 5 weeks for the BE CAR-33 therapy and the transplant will be scheduled 4 weeks after the end of BE CAR33. Patients will be monitored every month for the first three months and then every 6 months.

What are the possible benefits and risks of participating? Taking part in the study of testing 'ready-made' CAR T cells could help reduce the amount of disease and get the patient into remission before a planned bone marrow transplant. Leukaemia is less likely to come back after a bone marrow transplant if levels in the bone marrow are undetectable. The ready-made CAR T cells are being used to try and improve the chances of successful transplantation. Side effects may include low blood cell counts, infections, cytokine storm (severe immune reaction), graft versus host disease (where the donated cells attack the body) and other complications.

Being tested
Cryopreserved BE CAR33 T Cells (BE752TBTTBCAR33PBL)
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Cryopreserved BE CAR33 T Cells (BE752TBTTBCAR33PBL)Biological

    Also called BECAR33

    Single-dose intravenous infusion (weight-based dosing) of a banded dose of CAR33+ T cells/kg. BECAR33 Total duration of treatment: 28 days follow up: 12 months

Study groups in NCT05942599
Study groupRoleWhat they receive
Single dose intravenous infusion of a banded dose of CAR33+ T Cells/Kg BECAR33ExperimentalPatients will undergo careful screening to confirm that this treatment is appropriate for them. Patients will receive BE CAR-33 before their scheduled bone marrow transplant. Chemotherapy will be given prior to BE CAR-33 infusion to improve the ability of CAR T-cells to establish and grow. Patients will then receive a single infusion of the BE CAR-33 cells and will be closely monitored in hospital for the next 4 weeks. Patient will start chemotherapy for their scheduled bone marrow transplant 28 days after BE-CAR33 infusion unless their disease is progressing. Patients will be monitored on the study for 1 year after transplant and then long term in routine clinics.
Receives: Biological: Cryopreserved BE CAR33 T Cells (BE752TBTTBCAR33PBL)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Frequency and description of adverse events after BE-CAR33 Infusion

    Incidence of grade 3-5 toxicities occurring from infusion up to one year follow-up. Severe Adverse reactions of special interest will be CRS, ICANS, GvHD and VOD. American Society of Bone Marrow transplantation grading scales for CRS/ICANS, National institute of health criteria for GvHD and EBMT criteria for VOD will be applied. Common terminology criteria for adverse event (CTCAE) nomenclature will be used to grade other adverse events.

    Measured: 1 Year

Secondary outcomes (1)

  1. Number of patients achieving disease remission ahead of allo-SCT

    Remission rate will be assessed by bone marrow and CNS evaluation after 28 days. Disease remission is defined as morphological complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) with MRD by flow and/or PCR.

    Measured: 28 Days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted12 Jul 2023
  2. Study start21 Jul 2023
  3. Primary completion1 Jun 2026 (estimated)
  4. Study completion1 Jun 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Model description
Phase 1, open label, non-randomised
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05942599
Sponsor's protocol ID
18IC13
Acronym
CARAML
Status last verified
Jan 2026
First submitted
4 Jul 2023
Last updated
14 Jan 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing Cryopreserved BE CAR33 T Cells (BE752TBTTBCAR33PBL) in people with Relapsed Acute Myeloid Leukaemia, run by Great Ormond Street Hospital for Children NHS Foundation Trust at 1 site in United Kingdom.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 14 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Frequency and description of adverse events after BE-CAR33 Infusion (time frame: 1 Year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05942599.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Jan 2026.

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